Ocuvia shows promise in dry age-related macular degeneration (AMD) studies

BOSTON, MA, USA I February 13, 2015 I Stealth BioTherapeutics (Stealth), a clinical-stage biopharmaceutical company developing drug candidates for the treatment of diseases involving mitochondrial dysfunction, today announced the findings from several studies of Ocuvia in models of dry age-related macular degeneration (AMD). Ocuvia is an investigational drug that targets the inner mitochondrial membrane to treat eye disorders both common and rare, including retinal diseases and inherited mitochondrial optic neuropathies. The studies with Ocuvia were part of the Bascom Palmer Eye Institute annual Angiogenesis, Exudation, and Degeneration Conference in Miami.

Scott Cousins, M.D., Robert Machemer Professor of Ophthalmology and Director of the Duke University Center for Macular Diseases, presented nonclinical data with Ocuvia for dry AMD. Dr. Cousins is a retina-trained ophthalmologist specializing in the diagnosis and treatment of macular diseases, especially dry AMD. AMD is the leading cause of vision loss in older adults and more than 10 million people in the United States are affected by the disease.

Dr. Cousins showed that, in nonclinical models of dry AMD, Ocuvia prevented retinal cell damage, a hallmark of this debilitating disease. These results and emergent research underscore the role of mitochondrial dysfunction in dry AMD, a disease for which there is currently no FDA-approved treatment.

Mitochondria are the cell’s powerhouse, responsible for more than 90% of the energy our bodies need to sustain life and prevent disease. Preserving and restoring mitochondrial function in diseases of the eye represents a novel therapeutic approach for AMD, retinal vascular diseases and diabetic macular edema (DME) and retinopathy. In nonclinical studies, Ocuvia has been shown to modify retinal disease progression by targeting the inner mitochondrial membrane and restoring cellular energetics and function.

Dr. Cousins’s data highlighted the impairment of mitochondrial function in dry AMD, which was prevented by Ocuvia. “These are early, but exciting findings that Ocuvia helps prevent important and relevant pathological indicators of retinal injury in dry AMD, a disease that can over time cause profound blindness,” explained Dr. Cousins. “Mitochondrial dysfunction appears to be a core defect driving onset and progression of this disease, and these results suggest that preserving and restoring mitochondrial function may be a novel route to slowing dry AMD.”

Currently, Ocuvia is being studied in patients with dry AMD and DME in a clinical trial led by Jeffrey Heier, M.D., Director of the Vitreoretinal Service at Ophthalmic Consultants of Boston in Boston, Massachusetts. “Today, patients with dry AMD have no treatment options that address the manifestations of this growing disease,” said Dr. Heier. He added, “Ocuvia’s mechanism, coupled with the nonclinical work from Dr. Cousins and others, underscores its potential as a treatment for dry AMD and the role of mitochondrial dysfunction across diseases of the eye, especially those of the retina.”

“We are focused on pursuing macular diseases within our ophthalmology program in the hopes of providing patients with the first FDA-approved therapy for dry AMD,” said Chief Medical Officer Ben Bronstein. “We look forward to continuing our research and clinical studies for dry AMD with an aim toward offering a potential life-changing therapy to this underserved patient population.”

Mitochondria, The Cell’s Powerhouse

Mitochondria are the cell’s powerhouse, responsible for more than 90% of the energy our bodies need to sustain life and support growth. The energetics from mitochondria maintain healthy physiology and prevent disease. In many common and rare diseases, dysfunctional mitochondria are a key component of disease progression.

About Bendavia™ and Ocuvia™

Stealth’s lead candidates, Bendavia and Ocuvia, are investigational drugs with the potential to modify disease through mitoprotection—the ability to preserve energetics and restore normal energy production in mitochondria, while decreasing oxidative stress. These clinical candidates are being developed for both common and rare diseases including mitochondrial diseases where there are no FDA-approved treatments. The underlying science of Bendavia and Ocuvia is supported by more than 100 independent, peer-reviewed publications and abstracts. These mitochondrial-targeted candidates represent a novel therapeutic approach with the potential to address a wide variety of diseases having unmet treatment needs.

Stealth BioTherapeutics: Leading Mitochondrial Medicine

Stealth BioTherapeutics is a privately held biopharmaceutical company committed to bringing mitochondria therapies to patients to treat both common and rare diseases. As a key common element in a variety of serious, debilitating diseases, mitochondria – the cell’s energy source – offer a promising, and yet untapped, target to modify diseases with significant unmet treatment needs. Stealth is expanding its clinical development program to additional therapeutic areas, including cardio-renal diseases, ophthalmic disorders and orphan mitochondrial diseases. By defining the broad potential of its mitochondrial platform and therapies, Stealth is leading mitochondrial medicine.

SOURCE: Stealth BioTherapeutics